The first 60 days: Acute myeloid leukaemia in children
Acute myeloid leukaemia in children carries gene fusions rather than the mutations of ageing, is treated with four or five intensive courses of chemotherapy, and cures around two thirds of children. Adding gemtuzumab ozogamicin lowered relapse in the AAML0531 trial, and the menin inhibitor revumenib is the first targeted drug approved for the KMT2A-rearranged form common in young children. Below, week by week, is what OnCo's record of Acute myeloid leukaemia in children says about the first two months: the order is typical, the timing is yours to ask about. Sections appear only where the record has something to say. Orientation, not medical advice.
What happens now
Staging tests establish exactly what and where the cancer is. Everything else follows from the answers.
Ask which of these were tested and what the results were; see the report reader for what each value means.
These specialties appear in the standard of care for this cancer. In most centres they meet weekly as a tumour board to agree each plan; you can ask when yours was discussed and what was decided.
- PathologistNamed in the standard of care for: Induction (two courses), High-risk genetics or persistent residual disease.
- Medical oncologistNamed in the standard of care for: Induction (two courses), Consolidation (two or three courses), FLT3-ITD, Relapsed or refractory and 1 more.
- Transplant and cell therapy teamNamed in the standard of care for: Induction (two courses), Consolidation (two or three courses), High-risk genetics or persistent residual disease, Relapsed or refractory.
- Palliative and supportive care teamNamed in the standard of care for: Supportive care and late effects.
A second opinion from a centre that treats many similar cases is normal, not rude; the standard of care tells you what to compare it against.
Each row is a setting from the standard of care, in the order it usually arises. Not all will apply to you; your stage and biomarkers decide which do. The guideline grade, where recorded, says how strong the evidence is.
- 1.High-risk genetics or persistent residual diseaseNCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)
Allogeneic transplant in first remission.
- 2.Induction (two courses)NCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)
Cytarabine with daunorubicin or mitoxantrone and etoposide; gemtuzumab ozogamicin added to the first course for CD33-positive disease (AAML0531); MRD after course 1.
- 3.Consolidation (two or three courses)NCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)
High-dose cytarabine-based courses; no maintenance.
Sorafenib added to chemotherapy for high allelic ratio (AAML1031); gilteritinib in paediatric trials.
- 5.Relapsed or refractoryNCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)
Fludarabine and cytarabine reinduction; revumenib for KMT2A-rearranged disease; gilteritinib for FLT3; venetoclax combinations in trials; transplant.
- 6.Supportive care and late effectsNCI PDQ: Childhood Acute Myeloid Leukemia Treatment (health professional version)
Dexrazoxane cardioprotection with anthracyclines, transfusion and antimicrobial support, cardio-oncology follow-up.
Generated from this cancer's standard of care, biomarkers and open problems. Take the group that matches where you are. To tick, add your own and print, open the one-page appointment sheet.
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example Karyotype and FISH for recurrent fusions, RNA fusion panel, FLT3-ITD and allelic ratio, CD33 expression, Flow cytometry MRD after course 1 and course 2), and what were the results?These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Recognised subtypes for this cancer include Core-binding-factor AML, KMT2A-rearranged AML, FLT3-ITD AML.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Induction (two courses)
- For my situation (induction (two courses)), which of the standard options do you recommend and why?Guideline options include: Cytarabine with daunorubicin or mitoxantrone and etoposide; gemtuzumab ozogamicin added to the first course for CD33-positive disease (AAML0531); MRD after course 1.
- Am I a candidate for Cytarabine, Daunorubicin, Mitoxantrone or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of COG AAML0531 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Consolidation (two or three courses)
- For my situation (consolidation (two or three courses)), which of the standard options do you recommend and why?Guideline options include: High-dose cytarabine-based courses; no maintenance.
- Am I a candidate for Cytarabine, Etoposide, Mitoxantrone or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
FLT3-ITD
- For my situation (flt3-itd), which of the standard options do you recommend and why?Guideline options include: Sorafenib added to chemotherapy for high allelic ratio (AAML1031); gilteritinib in paediatric trials.
- Am I a candidate for Sorafenib, Gilteritinib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
High-risk genetics or persistent residual disease
- For my situation (high-risk genetics or persistent residual disease), which of the standard options do you recommend and why?Guideline options include: Allogeneic transplant in first remission.
Relapsed or refractory
- For my situation (relapsed or refractory), which of the standard options do you recommend and why?Guideline options include: Fludarabine and cytarabine reinduction; revumenib for KMT2A-rearranged disease; gilteritinib for FLT3; venetoclax combinations in trials; transplant.
- Am I a candidate for Fludarabine, Cytarabine, Revumenib or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of AUGMENT-101 apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Supportive care and late effects
- For my situation (supportive care and late effects), which of the standard options do you recommend and why?Guideline options include: Dexrazoxane cardioprotection with anthracyclines, transfusion and antimicrobial support, cardio-oncology follow-up.
- Am I a candidate for Dexrazoxane, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Any stage
- Are there clinical trials I could join, for example of Revumenib, Gilteritinib, Venetoclax, Ziftomenib?Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Supportive care improves quality of life and helps patients complete treatment.
- I read that “A third of children relapse and only half of those are cured”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “Infant AML with NUP98 or CBFA2T3::GLIS2 fusions has no effective therapy”. How does that affect my plan?Open problems are where trials and second opinions matter most.
Trials open now for this cancer in OnCo, largest phase first. Joining a trial is a decision like any other: ask what the comparison arm is, whether a placebo is used, and what happens if you leave. The cancer page searches ClinicalTrials.gov live for more.
- A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AMLPhase 2 · active · NCT03591510A Phase II, Open-label, Single Arm Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Twice Daily Midostaurin (PKC412) Combined With Standard Chemotherapy and as a Single Agent Post-consolidation Therapy in Children With Untreated FLT3-mutated AML
- Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid TumorsPhase 1/2 · recruiting · NCT03934372An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Ponatinib for the Treatment of Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors in Pediatric Participants
- Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the BloodPhase 1/2 · active · NCT03793478A Phase 1/2, Multicenter, Dose-Escalating Study To Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy Of Quizartinib Administered in Combination With Re-Induction Chemotherapy, and as a Single-Agent Continuation Therapy, in Pediatric Relapsed/Refractory AML Subjects Aged 1 Month to <18 Years (and Young Adults Aged up to 21 Years) With FLT3-ITD Mutations
- Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell TransplantPhase 1/2 · active · NCT03301168Phase I/II Study of CaspaCIDe® T Cells From an HLA-Partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders
Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.
- Acute myeloid leukaemia in children: the full pageAcute myeloid leukaemia in children carries gene fusions rather than the mutations of ageing, is treated with four or five intensive courses of chemotherapy, and cures around two thirds of children. Adding gemtuzumab ozogamicin lowered relapse in the AAML0531 trial, and the menin inhibitor revumenib is the first targeted drug approved for the KMT2A-rearranged form common in young children.
- One-page appointment sheetYour questions, the words you may hear, what to bring, and space for the answers. Print it.
- Treatment sequencingWhich treatment tends to follow which, line by line.
- NavigatorStandard of care for your stage, what you have tried, and trials near you.
- Secondary malignancy (therapy-related cancer): A new, different cancer caused by the treatment of the first one: leukaemia after alkylating chemotherapy or PARP inhibitors, solid tumours in irradiated tissue decades later, and rarely T-cell lymphoma after CAR-T.
- Late effects and survivorship toxicity: Health problems appearing months or decades after treatment ends: heart damage, infertility, second cancers, lymphoedema, dry mouth, memory problems, weak bones.
- Minimal / molecular residual disease (MRD): Cancer still present after treatment but too small to see on scans, detected by blood or marrow tests.
Every term links to the glossary.